Solid Biosciences has received clearance from the U.S. Food and Drug Administration (FDA) for an Investigational New Drug (IND) application for SGT-003, their microdystrophin gene therapy candidate for the treatment of Duchenne muscular dystrophy.

What is SGT-003?

SGT-003 combines a differentiated microdystrophin transgene with a new, novel capsid (the protein shell of the virus used to deliver the drug) designed to substantially increase the amount of drug delivered to muscle compared to first-generation capsids. In pre-clinical studies, this has resulted in higher levels of microdystrophin expression and greater improvements in muscle function. In addition, SGT-003 is manufactured using an improved process to maintain a high level of full capsids and overall drug purity.

“We are pleased to have the FDA’s clearance to proceed into the clinic with SGT-003, a new, innovative gene therapy candidate for Duchenne.”

Bo Cumbo, President and CEO at Solid Biosciences

The IND clearance enables Solid to begin enrolling patients in INSPIRE DUCHENNE, a Phase 1/2 multicenter clinical trial to primarily evaluate the safety and tolerability of SGT-003. The study will include two separate cohorts based on age, beginning with patients 4 and 5 years old. All participants will receive a one-time intravenous infusion of SGT-003 at a single dose level. Long-term safety and efficacy will be evaluated for a total of five years following treatment.

Defeat Duchenne Canada looks forward to working with SOLID to bring the INSPIRE DUCHENNE study to Canada.